Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

In boys with DMD, executive skills such as self-control begin to lag around age 8, but show signs of partial catch-up by 14, ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort. Delandistrogene ...

The Muscular Dystrophy Association (MDA) is proud to announce the recipients of the 2026 MDA Legacy Awards, recognizing excellence in scientific and clinical research and saluting outstanding ...

In research published in Developmental Medicine & Child Neurology , investigators developed a brief, reliable, and valid screening tool to help ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

High adherence rates to PMO therapies were observed in patients with DMD, indicating patient commitment to treatment regimens. Significant challenges exist in using claims data to assess PMO treatment ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...

When many people think of the Muscular Dystrophy Association (MDA), they probably think about the long-running telethon made famous by comedian Jerry Lewis. For decades, the telethon took over the ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...